A synthetic molecule may help fight disease

University of Missouri researchers have found that synthetic molecules might prove to be a key in reducing the severity of Spinal Muscular Atrophy, or SMA, the leading genetic cause of infantile death.

The work is led by Chris Lorson, a researcher at the Bond Life Sciences Center and a professor in both the Department of Veterinary Pathobiology and the Department of Molecular Microbiology and Immunology.

SMA is a genetic disease inherited by one in 6,000 children who are missing a gene, SMN-1, that produces a protein needed to direct spinal nerves to command muscles. Humans have a partially functioning backup gene, SMN-2, that makes a small amount of the needed protein.

Lorson’s team introduced synthetic ribonucleic acid — a biologically fundamental molecular similar to DNA — into mice carrying genes responsible for the disease. Doing so, they found, triggered the backup gene and lowered the severity of SMA.